MyoKardia is dedicated to revolutionizing the treatment of cardiovascular disease. The company's first programs include hypertrophic and dilated cardiomyopathy (HCM and DCM), which together afflict approximately 1 million people in the United States, and for which no novel therapeutics have been brought to market in over a decade.
Targeting Disease at the Source
MyoKardia's proprietary drug discovery platform brings together advances from the fields of cardiovascular genomics and heart muscle biology to enable its scientists to target certain heart disease 'at its source.' Abnormalities in the basic unit of heart muscle, called the sarcomeres, have been identified as the driving cause for a variety of heart disease, and the most common cause of hypertrophic and dilated cardiomyopathy. Mutations in the proteins of the sarcomere cause disease by rendering the muscle either 'hyper' or 'hypo' contractile.
Developing Personalized Therapeutics & Employing Small Drug-Like Molecules
MyoKardia's platform allows for the rapid development of mutation-specific sarcomeric allosteric modulators that rebalance contractility, therefore stopping and potentially reversing the course of disease. The company's initial focus is developing a pipeline of novel, small molecule therapeutics for HCM and DCM.
Long-term Vision that Addresses Complex Heart Failure
This genetically targeted approach has the potential to revolutionize the treatment of cardiomyopathies, and ultimately a broader spectrum of cardiovascular disease, including heart failure.
MyoKardia is a private company founded in 2012 by Third Rock Ventures and world-leading scientific researchers known for their expertise in muscle biology and cardiovascular disease genetics.